Today, the National Basic Medical Insurance, Work-related Injury Insurance and Maternity Insurance Drug List (2024) (hereinafter referred to as the "New Medical Insurance List") was officially announced, and the world's first and only ROCK2 inhibitor Ilex® (besudinil mesylate tablets) approved for use in patients aged 12 years and above with chronic graft-versus-host disease (cGVHD) who have an inadequate response to glucocorticoid treatment was included in it . The adjusted new version of the medical insurance drug list will be officially implemented on January 1, 2025.

01. Chronic rejection after transplantation: the hidden “killer” and the dilemma of fibrosis

cGVHD is the most common and serious complication after allogeneic hematopoietic stem cell transplantation, with an incidence of 30%-70%, and is the main cause of late non-relapse death two years after transplantation. The complexity of cGVHD lies in its invasion of multiple organs - from the skin to the lungs, from the eyes to the joints, covering many organs. This "systemic attack" not only seriously affects the patient's long-term quality of life, but also brings a heavy economic and living burden.

In terms of the "root cause", fibrosis is the main problem of cGVHD and the core difficulty in treatment. The treatment goal of cGVHD is to establish immune tolerance to maintain immune homeostasis, thereby effectively preventing and reducing the process of fibrosis. On the other hand, for tissues and organs that have already undergone fibrosis, it is hoped that fibrosis can be effectively reversed. Therefore, restoring immune homeostasis and reversing fibrosis are the core goals of cGVHD treatment.

Currently, more than half of cGVHD patients still need second-line or later-line treatment after receiving first-line standard treatment. Patients need more targeted treatments to reverse the fibrosis dilemma.

02. Breakthrough and turning point: ROCK2 inhibitors help patients reverse the fibrosis dilemma

With the continuous progress and breakthroughs in medical technology, the treatment of cGVHD has also entered the era of targeted therapy. Targeted therapy represented by ROCK2 inhibitors has opened up an innovative treatment path for patients. The unique and innovative mechanism of action of ROCK2 inhibitors can simultaneously target immune inflammation and fibrosis, reduce or even reverse fibrosis while establishing immune homeostasis, and help patients achieve treatment goals and standardized management as early as possible.

The Chinese Society of Clinical Oncology (CSCO) and the Hematology Branch of the Chinese Medical Association have successively released and updated authoritative guidelines and consensus for the treatment of chronic graft-versus-host disease (cGVHD). Among them, besudilide mesylate is the only newly recommended drug included in the second-line treatment, providing a new treatment option for addressing the problem of fibrosis in the treatment of cGVHD.

In addition to the core difficulty of fibrosis, another major problem facing cGVHD is delayed diagnosis and treatment. In the past, doctors and patients paid more attention to recent complications such as engraftment, acute rejection, infection, and recurrence of primary malignant blood diseases in the postoperative management of allogeneic hematopoietic stem cell transplantation. Because the early symptoms of cGVHD are not obvious, patients often tend to ignore the occurrence of this complication, resulting in 50% of cGVHD patients being moderate to severe at the time of diagnosis, which not only increases the difficulty of treatment, but also brings an extremely heavy disease burden to patients.

Multiple II studies at home and abroad have shown that in patients with moderate to severe cGVHD who have failed multiple lines of treatment, the best overall response rate (ORR) of besudiol mesylate was 73.3%-76%, and it was well tolerated. In patients who started besudiol mesylate treatment earlier after cGVHD diagnosis, the ORR was as high as 83%, indicating that earlier intervention can bring more benefits to patients.

This is not only a benefit of earlier treatment, but also a process of standardized management after transplantation. In recent years, the average annual growth rate of allogeneic hematopoietic stem cell transplantation in my country has reached 10%. In 2023, 216 medical institutions in my country completed a total of 14,952 allo-HSCTs, ranking first in the world. In response to the unmet needs of standardized management, the discipline construction around cGVHD is constantly deepening. With the standardized management model of follow-up after hematopoietic stem cell transplantation as the core, key nodes are empowered, the national diagnosis and treatment network is accelerated, regional coordinated development is promoted from point to surface, and interdisciplinary collaboration is further optimized. This patient-centered model not only improves treatment efficiency, but also provides valuable experience for the management of other transplant complications.

03. Leveraging the future: Sanofi’s global transplant layout and clinical ambitions

The rapid inclusion of Besudil Mesylate Tablets in the new version of the medical insurance catalog is due to Sanofi's deep insight into the market and strategic layout. In the third quarter of 2024, its global sales increased by 57.8% year-on-year, and the Chinese market became one of the important engines driving growth.

At the same time, the global multicenter Phase III clinical trial of besudiol mesylate tablets combined with prednisone for the first-line treatment of cGVHD and the global multicenter Phase III clinical study of besudiol mesylate tablets for the treatment of chronic allograft lung function loss (CLAD) after adult bilateral lung transplantation are being promoted simultaneously. With the deepening of research, it is expected to further expand from the second-line treatment of cGVHD to the first-line treatment, becoming a benchmark drug in the field of cGVHD treatment. It also marks the further exploration of ROCK2 inhibitors from blood transplantation to solid organ transplantation, providing new treatment options for a wider range of patient groups.

With the successful inclusion of Sanofi's Besudinil Mesylate Tablets in the medical insurance catalogue, the growth potential of ROCK2 inhibitors will be even greater. At the same time, Sanofi is also promoting more research and development and diversified innovative cooperation, and is expected to usher in a new era in the field of transplantation.

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